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1、2022 Clinical Trials RoundupOctober 2023White PaperHeidi ChenAssociate Director,Research&Commercial Services2October 2023 Copyright 2023 Citeline,a Norstella company.(Unauthorized photocopying prohibited).2022 Clinical Trials Roundup1.Citeline(2022)2021 Clinical Trials Roundup:Clinical Trials Resili

2、ence After Pandemic Disruptions.2.The data snapshot dates for full-year activity are June 26,2023;June 23,2022;June 14,2021;June 12,2020;June 5,2019;June 6,2018;and July 6,2017.Due to delays in reporting of trial activity,counts for prior years will have likely grown since the original data snapshot

3、 date.Last year we remarked on the resilience and return of clinical trial momentum post-pandemic,where we saw a 6%growth of total trials(22%excluding COVID-19 trials)in 2021.1 One year later,the roller coaster resumes as the healthcare industry continues to be influenced by the global economy and p

4、olitical climate.Big headline events the Inflation Reduction Act,the ongoing Russo-Ukrainian war,harsh funding cuts and divestments in the biopharma sector,and stalled economic growth in China are just a few headwinds the healthcare industry braved in 2022.As the world picks up the pace to rebuild m

5、omentum and put the pandemic behind it,navigating through the evolving regulatory guidance and market dynamics calls for endurance and perseverance.As with each edition of our Clinical Trials Roundup,we focus on an overview of the Phase IIII clinical trials that initiated in the prior calendar year(

6、2022)across all therapeutic areas comprehensively covered by Trialtrove,as well as in-depth analyses into the key diseases,players,and geographies that impact the pharmaceutical industry.We will also look at any lingering effect of the pandemic,recovery,and challenges of adapting to the new equilibr

7、ium.As of June 26,2023,Trialtrove curated 9,104 Phase IIII clinical trials(Table 1)investigating at least one drug and with a disclosed start date within the calendar year of 2022,returning a decline of 12.5%,breaking the upward trend of clinical trial counts for the first time since 2016.2 In 2021,

8、we saw a year-on-year(Y-O-Y)growth rate that returned to a more comparable level to pre-pandemic years,once the COVID disruptions appeared to have slowed down.If we exclude COVID-19 trials from the 2021 mix,trial growth returned a remarkable 22%.In 2022,there are many fewer trials overall;even with

9、the exclusion of COVID-19 trials,trial initiations still exhibited a decline of 6%.The clinical trial landscape changes a bit if we take a closer look at industry-sponsored trials,which may better represent the current health of the pharmaceutical industry.In 2022,industry-sponsored trials(Table 2)d

10、ecreased by 7%overall,but if we exclude COVID-19 trials,this reduces to a 4%decline,reflecting the industrys survival mode since 2021.Introduction3October 2023 Copyright 2023 Citeline,a Norstella company.(Unauthorized photocopying prohibited).2022 Clinical Trials RoundupYear of trial initiation20222

11、02120202019201820172016Trial count9,10410,4109,8197,7657,6066,7946,067Year-on-year growth(%)-12,50%6%26%2%12%12%N/ATrial count (excluding COVID-19 trials)8,5419,0777,4247,7657,6066,7946,067Year-on-year growth (excluding COVID-19 trials,%)-6%22%-4%2%12%12%N/AYear of trial initiation202220212020*2019*

12、2018*2017*2016*Industry sponsored trials6,1516,6466,5426,2116,1275,6845,089Year-on-year growth(%)-7%2%5%1%8%12%N/AIndustry sponsored trials(excluding COVID-19 trials)5,8076,0275,7096,202-Year-on-year growth (excluding COVID-19 trials,%)-4%6%-8%N/A-Table 1:Phase IIII clinical trial activity by volume

13、 and growth,201622Table 2:Phase IIII industry-sponsored trials,201622 Source:Trialtrove,June 2023*Data accessed August 2023 Source:Trialtrove,June 2023The data in Table 2 also support a trending departure of COVID-19 trials research by industry sponsors in 2021(619 trials)and 2022(344 trials).Compan

14、ies such as Roche and Novartis have shifted their focus away from COVID-19 over the last two years,while Pfizer held on like a long-distance champion in this arena.The exclusion of COVID-19 trial count is a way to minimize the COVID effect on the trials landscape,providing a surrogate health check o

15、n clinical research.The decrease in trial numbers in 2022 demonstrated a more restrained and cautious environment for clinical research,guided by a complex myriad of factors:the global economy,legislation,and the overall political climate.4October 2023 Copyright 2023 Citeline,a Norstella company.(Un

16、authorized photocopying prohibited).2022 Clinical Trials Roundup1641697231,0291,2281,3971,7113,3941581758511,0541,2611,4172,7713,78401000200030004000GenitourinaryOphthalmologyCardiovascularMetabolic/EndocrinologyAutoimmune/InflammationCNSInfectious DiseaseOncologyTrial count20212022Clinical Trial Ac

17、tivity by Therapeutic AreaThe ranking of therapeutic areas(TAs)by trial initiations in 2022 remained the same as in prior years,albeit most TAs saw a modest decrease in trial counts.Infectious disease(ID)had a dramatic rise to second place when its trial initiations rose from 760 trials in 2019 to 3

18、,053 trials in 2020,largely contributed by COVID-19 trials.While the pandemic persisted through 2021,the number of ID trial initiations dropped to 2,771 trials,and further decreased to 1,711(-38%)in 2022,closing the gap of its lead over central nervous system(CNS)trials.Oncology(Onc)continued to be

19、the top-ranking TA with a clear lead,even though its trial initiations were down by 10%.The cardiovascular TA experienced a 15%decrease of trial initiations in 2022,while other TAs such as metabolic/endocrinology(Met),autoimmune/inflammation,ophthalmology(Oph),and CNS held on to their 2021 rebound a

20、nd saw a smaller reduction between 1%and 3%.Genitourinary(GU)is the only TA with growth,posting a 4%rise in 2022.Figure 1:Phase IIII clinical trial initiations by therapeutic area3 Source:Trialtrove,June 20233.Trials that include multiple indications across different TAs will be counted for each tar

21、geted TA.As such,the sum of trial counts for the eight TAs will be higher than the total number of Phase IIII trials started in 2022.Trial counts for ID include activity from vaccines(infectious diseases),which is a separate TA module within Trialtrove.For the purposes of this analysis,all ID activi

22、ty has been combined into a single TA.5October 2023 Copyright 2023 Citeline,a Norstella company.(Unauthorized photocopying prohibited).2022 Clinical Trials RoundupTrial Phase DistributionsClinical trial initiations saw a similar trend across Phase IIII;Phase I had the most initiations,followed by Ph

23、ase II and Phase III.There was an increase in Phase I trials(+4%),which nearly matched the decrease in Phase II trials(-3%).The proportion of mixed phases(Phase I/II and Phase II/III)and Phase III trials had little to no change.The current phase distributions reflect a healthy return to new research

24、 activities and robust pipelines in development.Figure 2:Distribution of Phase IIII clinical trials by trial phase,202122 Source:Trialtrove,June 202337%9%34%4%17%41%9%31%3%16%0%5%10%15%20%25%30%35%40%45%Phase IPhase I/IIPhase IIPhase II/IIIPhase III2021(n=10,410)2022(n=9,104)6October 2023 Copyright

25、2023 Citeline,a Norstella company.(Unauthorized photocopying prohibited).2022 Clinical Trials Roundup2552752933433503673924825635660100200300400500600700Pain(nociceptive)Respiratory VaccinesColorectalRespiratory InfectionsType 2 DiabetesLymphoma,Non-HodgkinsBreastLung,Non-Small CellNovel coronavirus

26、(2019-nCoV,COVID-19)Unspecified Solid Tumor2022Phase IPhase I/IIPhase IIPhase II/IIIPhase IIITop Diseases for Phase IIII Clinical TrialsEach year we analyze the top 10 diseases for clinical trial activities to get a glimpse of where research efforts are taking place.After a two-year reign,COVID-19 f

27、inally gave up its number one spot back to an oncology disease(unspecified solid tumor,566 trials),though it continues to exert its presence in a close second place(563 trials).Clearly,the pandemic disruptions to clinical trials have subsided.The makeup of the top 10 diseases has been consistent for

28、 several years,with some slight ranking shifts.Oncology diseases make up half of the top 10 chart,and four out of five diseases are within the top five:unspecified solid tumor,non-small cell lung cancer(NSCLC),breast cancer,and non-Hodgkins lymphoma(NHL).The COVID-19 trials clearly had a major cut f

29、rom 1,333(13%)in 2021 to 563(6%)in 2022.However,the phase distribution of COVID-19 trials in 2022 is more evenly distributed between Phases I,II,and III,as opposed to the inflated Phase II trial numbers seen in prior years.Respiratory vaccines and respiratory infections were the other two indication

30、s that rode the wave with the COVID-19 trials,but their numbers also took a dive and moved down the ranks in 2022.Head and neck cancer dropped off the top 10,making way for pain(nociceptive)to rejoin the fold as number 10.Most of the other top diseases had a modest reduction of trial initiations,tho

31、ugh their proportion with respect to total trials barely changed.Type 2 diabetes is the disease within the top 10 that had the highest increase in trial initiations,exhibiting rising activity in early-phase development.Figure 3:Top 10 diseases in Phase IIII clinical trial initiations,202122 Source:T

32、rialtrove,June 20237October 2023 Copyright 2023 Citeline,a Norstella company.(Unauthorized photocopying prohibited).2022 Clinical Trials Roundup05001,0001,5002,0002,5003,0003,500201120122013201420152016201720182019202020212022Trial countYearThe steady upward trend of rare disease R&D also suffered a

33、 setback in 2022,with 13%fewer trial initiations than the previous year,wiping away the post-pandemic rebound observed in 2021(Figure 4).Figure 4:Clinical trials in rare diseases over the last 12 years Source:Trialtrove,June 20232021Phase IPhase I/IIPhase IIPhase II/IIIPhase III286314314374405447487

34、5415751,33302004006008001,0001,2001,400Head/NeckColorectalType 2 DiabetesBreastLymphoma,Non-HodgkinsRespiratory InfectionsRespiratory VaccinesLung,Non-Small CellUnspecified Solid TumorNovel coronavirus(2019-nCoV,COVID-19)Source:Trialtrove,June 20238October 2023 Copyright 2023 Citeline,a Norstella co

35、mpany.(Unauthorized photocopying prohibited).2022 Clinical Trials Roundup405246286207252214367227223207206195050100150200250300350400450NHLEsophagealHead/NeckPancreasLiverOvarian20212022The receding trial initiations in 2022 no doubt impact rare disease trials.The nature of rare disease research inh

36、erently faces tougher challenges,as most conditions are debilitating or fatal,particularly in pediatrics.Due to a limited patient pool for rare diseases,clinical trials often had to expand recruitment to multiple countries for suitable patients,resulting in longer trial timelines and higher expendit

37、ure.The depressed economic climate for biopharma in 2022 certainly accentuated the challenges further.However,this does not eliminate the progress made by patient advocacy groups and regulators that aim to bring patient-centric pathways to rare disease research.The US Food&Drug Administration(FDA)ro

38、llout of patient listening sessions gave rare disease patients a voice with a regulatory perspective.This effort was further complemented by publishing draft guidance documents on clinical endpoints that reflect patient voice.Amid a challenging year for biopharma,one can still celebrate success stor

39、ies such as Amylyxs newly launched amyotrophic lateral sclerosis(ALS)therapy and Relyvrios outstanding performance.4 Rare disease research seems to be picking up again or at least sustaining in 2023,as 1,439 clinical trials have already initiated to date.5Historically,oncology indications have domin

40、ated the rare disease landscape,and 2022 is no different(Figure 5).NHL continues to be the most studied rare disease,while other indications experienced minor decreases in trial counts.Only head and neck and liver cancer experienced notably fewer trial starts in 2022.Outside oncology,the top three r

41、are indications in 2022 were the same as in the previous year:ALS(35),tuberculosis(33),and sickle cell disease(28).Figure 5:Top indications in rare diseases,by trial initiations,202122 Source:Trialtrove,June 20234.Scrip(May 12,2023).Amylyxs ALS Treatment Relyvrio Beats Expectations Again.5.Rare dise

42、ase clinical trials search output from Trialtrove,August 2023.9October 2023 Copyright 2023 Citeline,a Norstella company.(Unauthorized photocopying prohibited).2022 Clinical Trials RoundupTop industry leaders of the clinical trials landscape have largely remained consistent each year,with minor shuff

43、ling of the rankings.The top 10 sponsors in 2022 happen to be the same cast as the previous year.Merck&Co.climbed up from third place in 2021 to unseat AstraZeneca from the No.1 spot and push it down to second.Roche follows right after AstraZeneca,moving from second to third place.Rounding up the to

44、p five again are Pfizer and Jiangsu Hengrui,though their rankings are switched from the previous year.Novartis and Johnson&Johnson(JNJ)also switched rankings,while Bristol Myers Squibb(BMS),Eli Lilly,and GlaxoSmithKline(GSK)kept the same placements.Key Industry Sponsors in the Trial LandscapeSponsor

45、2022 Trials2022 Ranking2021 Trials2021 RankingMerck&Co.14811513AstraZeneca12221721Roche11431622Pfizer11241225Jiangsu Hengrui 10651304Novartis9261217Johnson&Johnson8571226Bristol-Myers Squibb8481208Eli Lilly799809GlaxoSmithKline70107610Table 3:Ranking of top 10 industry trial sponsors by trial volume

46、s Source:Trialtrove,June 2023Just about all the top sponsors had fewer trial initiations in 2022,but AstraZeneca(-50 trials)and Roche(-48 trials)had the largest reductions compared to 2021(Figure 6).AstraZeneca had a much smaller number of Phase II trials(39 vs.64 in 2021)but maintained a similar le

47、vel of late-stage research in Phase III(33 vs.36).Roche saw significantly fewer trials in both Phase II and Phase III development.Pfizer gained some momentum in Phase II trials(38 vs.27 in 2021)as some of the early-phase trials advanced to mid-and late-phase trials.Jiangsu Hengrui,on the other hand,

48、kept the same volume of Phase I trials,while it engaged in fewer Phase IIIII trials.BMS initiated fewer trials through all phases,particularly in Phase III,where it had the fewest trial initiations among the top 10 sponsors.Eli Lillys increase in early-phase development made up the losses seen in Ph

49、ase II and Phase III,coming out near even compared with last year.GSK is the top sponsor that had the fewest changes of trial starts across phases.10October 2023 Copyright 2023 Citeline,a Norstella company.(Unauthorized photocopying prohibited).2022 Clinical Trials RoundupFigure 6:Top 10 industry sp

50、onsors by trial distributions from Phase IIII,202122 Source:Trialtrove,June 20232022Phase IPhase I/IIPhase IIPhase II/IIIPhase IIITotal7079848592106112114122148020406080100120140160GlaxoSmithKlineEli LillyBristol-Myers SquibbJohnson&JohnsonNovartisJiangsu HengruiPfizerRocheAstraZenecaMerck&Co.2021Ph

51、ase IPhase I/IIPhase IIPhase II/IIIPhase IIITotal7680120121122122130151162172020406080100120140160180200GlaxoSmithKlineEli LillyBristol-Myers SquibbNovartisJohnson&JohnsonPfizerJiangsu HengruiMerck&Co.RocheAstraZeneca11October 2023 Copyright 2023 Citeline,a Norstella company.(Unauthorized photocopyi

52、ng prohibited).2022 Clinical Trials RoundupTop Sponsor Disease PrioritizationA further dive into the top diseases of the most active sponsors reflects the trend of clinical research direction and activity of the industry.Between 2019 and 2021,the number of discrete diseases reduced from 22 down to 1

53、7.But 2022 seemed to reverse this trend,and the number of distinct top diseases increased to 20,reflecting a slightly more diverse portfolio.Coincidentally,six out of the top 10 companies kept their No.1 priority unchanged between 2021 and 2022 the same number of companies as in the previous edition

54、 of our Clinical Trials Roundup.Currently,most of the world views the pandemic as a past event.Some of the top sponsors have departed from COVID-19 clinical research since 2020;even JNJ and GSK have shifted their focus away from it,leaving Pfizer steadfastly committed in this arena.Other top priorit

55、ies for Pfizer included the related indications of respiratory infections and respiratory vaccines.Oncology indications in general have been the most studied TAs in clinical trials.Companies such as Merck&Co.,AstraZeneca,Roche,and BMS all kept a robust oncology portfolio in both years(Table 4).NSCLC

56、 remained a top priority among the most active industry sponsors,namely for Merck&Co.,AstraZeneca,and BMS.In 2022,Roche and Novartis also lifted NSCLC to their frontline focus.Unspecified solid tumor has remained a top priority for oncology-focused companies Jiangsu Hengrui and BMS,joined this year

57、by Merck&Co.However,this indication exited the top three for Roche and Novartis in 2022.Breast cancer is another highly studied disease within the top three for the most active companies this past year,though Pfizers focus on respiratory vaccines displaced it.In addition to Pfizer,Eli Lilly and GSK

58、are the only top companies that did not have an oncology disease in their top three chart.In fact,both Pfizer and GSK stayed loyal to their commitment in the respiratory indications,while Eli Lilly retained its focus on diabetes and related cardiometabolic comorbidities.Few ventured out to diversify

59、 their top priorities,except for JNJ,which stood out with its top three diseases spread across oncology,CNS,and autoimmune portfolios.12October 2023 Copyright 2023 Citeline,a Norstella company.(Unauthorized photocopying prohibited).2022 Clinical Trials RoundupTop diseases by trial volumeCompanyYear#

60、1#2#3Merck&Co.2022Unspecified Solid Tumor(18)Colorectal(15)NSCLC(13)2021NSCLC(16)Melanoma(13)Head/Neck(11)Renal Cancer(11)AstraZeneca2022NSCLC(18)Breast(10)COPD(7)2021NSCLC(30)NHL(16)Renal Disease(12)Breast(12)Roche2022NHL(14)NSCLC(11)Liver(9)2021Breast(19)NHL(18)Unspecified Solid Tumor(16)Pfizer202

61、2COVID-19 (20)Respiratory Infections(15)Respiratory Vaccines(10)2021COVID-19 (24)Respiratory Infections(13)Breast(10)Jiangsu Hengrui Pharmaceuticals2022Unspecified Solid Tumor(18)Breast(13)Pain(nociceptive)(12)2021Unspecified Solid Tumor(12)Pain(nociceptive)(9)Type 2 Diabetes(8)Breast(8)Prostate(8)N

62、ovartis2022Breast(13)NSCLC(8)Lupus(4)2021NSCLC(10)Breast(10)Dyslipidemia(9)Multiple Sclerosis(5)Chronic Myelogenous(5)Unspecified Solid Tumor(5)Johnson&Johnson2022Multiple Myeloma(13)Depression(7)Psoriasis(5)2021Multiple Myeloma(14)Respiratory Vaccines(14)Respiratory Infections(12)Crohns Disease(9)C

63、OVID-19(9)Bristol-Myers Squibb2022Unspecified Solid Tumor(11)NSCLC(8)Renal Cancer(8)2021NSCLC(13)Melanoma(11)Unspecified Solid Tumor(9)Multiple Myeloma(9)Eli Lilly2022Type 2 Diabetes(13)Obesity(10)Dyslipidemia(7)2021Type 2 Diabetes(13)Obesity(11)Breast(9)GlaxoSmithKline2022Respiratory Infections(13)

64、Respiratory Vaccines(10)HIV(7)2021Respiratory Vaccines(16)COVID-19(15)Respiratory Infections(11)Table 4:Top diseases by the most active sponsors,2022 vs.2021 Source:Trialtrove,June 202313October 2023 Copyright 2023 Citeline,a Norstella company.(Unauthorized photocopying prohibited).2022 Clinical Tri

65、als RoundupLocation Matters:Geographic Survey of Trial ActivityWe now tune in to location insights of trial initiations,revealing sweet spots and pain points.China retained its lead in trial initiations with 3,405 trials(vs.3,795 in 2021),with the US in a close second with 2,876 trials(vs.3,310 in 2

66、021)(Figure 8).In 2022,we observed a downward trend in trial starts across all regions,with the sharpest decline in Asia(-12%)(Figure 7).We have not yet seen the impact of pain points with the new EU Clinical Trials Information System(CTIS)on trial initiations,as that was only made mandatory by the

67、European Medicines Agency(EMA)Clinical Trials Regulation on Jan.31,2023.Although frustrations were heard loud and clear as sponsors struggled with CTIS earlier this year,it would be premature to project how this episode might interfere with trial activities in 2023 and the implications this may have

68、 on regional differences.Figure 7:Trial volumes by region,202122-14%-12%-10%-8%-6%-4%-2%0%-2,000-1,00001,0002,0003,0004,0005,0006,0007,000AsiaAmericasNorth AmericaEuropeWestern EuropeEastern EuropeAustralia/OceaniaWestern Asia/Middle EastSouth AmericaAfrica20222021Delta%Source:Trialtrove,June 202314

69、October 2023 Copyright 2023 Citeline,a Norstella company.(Unauthorized photocopying prohibited).2022 Clinical Trials RoundupA closer look at the country-level data reveals a rather consistent proportion of total trials.All countries had fewer trials in 2022,reflecting a shared trend of lower trial v

70、olume all around.Figure 8 compares the countries within the vicinity of the Russo-Ukrainian war,inclusive of Russia and Ukraine.Clearly Ukraine suffered the biggest loss of trial initiations(-154%)due to displacements of trial subjects,and clinical trials in Russia also plunged by 86%compared to 202

71、1.6 The lack of stability caused by the war extended to Poland and Hungary,where clinical trials went down by 35%and 34%,respectively.Both Poland and Ukraine were considered as up-and-coming locations for clinical trials in recent years as these countries made it to the top 10 chart for selected the

72、rapeutic areas with steady growth of trial activities.One can expect the low numbers of clinical trials in these regions to continue in 2023 as there has been no sign of armistice.Figure 8:Country-level trial numbers for China,US,and countries near the Russia-Ukraine conflict Source:Trialtrove,June

73、202320222021%changeChinaUSPolandRussiaCzechRepublicHungaryTurkeyUkraine20223,4052,8763932352202161828320213,7953,310529437274290214211%change-11%-15%-35%-86%-25%-34%-18%-154%2022%37%32%4%3%2%2%2%1%2021%36%32%5%4%3%3%2%2%-170%-150%-130%-110%-90%-70%-50%-30%-10%10%05001,0001,5002,0002,5003,0003,5004,0

74、006.Pink Sheet(April 6,2023).Russian Clinical Trials Plunge to Lowest Point in 19 Years Following Ukraine War.15October 2023 Copyright 2023 Citeline,a Norstella company.(Unauthorized photocopying prohibited).2022 Clinical Trials RoundupTable 5 summarizes the top 10 countries by trial volumes for the

75、 last two years,starting with the overall(all TAs)landscape,followed by a deeper dive into each TA.A quick survey across the TAs reflects fewer trials in 2022 and a consistent pattern of China and US being the top two locations with clear leads in all therapy areas,where China takes the No.1 spot in

76、 all but the CNS and Oph TAs.Overall,Autoimmune/Inflammation and Onc data shared a similar narrative,where the same top 10 countries remained relatively unchanged with minor shuffling of rankings between fifth and ninth places.When we compared trial volumes by TA earlier(Figure 1),ID had 38%fewer tr

77、ials than in 2021,which translates to a drop of over 1,000 trials.The fact that China held on to the zero-COVID policy for much of 2022 saw it take the lead in ID trials.Interestingly,Australia is the only country that saw a slight increase in ID trials and climbed up from 18th place in 2021 to fift

78、h place in 2022.This upward movement was likely inflated by the fact that most of the major markets had substantially cut back on COVID-19 trials.ID trials in the US,India,and Japan had the most reduction in trial volume.As we move towards post-pandemic equilibrium,it is not surprising to see the ge

79、ographical shift in ID trials.Top 10 ID locations welcomed new joiners such as Australia,Germany,and South Africa,displacing Iran,Brazil,Canada,and Russia.CNS had a relatively stable year,where growth in Australia,France,and Italy offset some of the volume loss,yielding a net reduction of 1%overall.

80、Pain(nociceptive)resides within the CNS TA,and it returned to the top diseases(Figure 3)after the disruptive pandemic years.In addition to pain(nociceptive),most CNS trials were devoted to depression,epilepsy,and Alzheimers disease(data not shown).Cardiovascular and Met have seen quite the shuffle o

81、f country rankings in the latter half of the top 10.Russia made its only appearance in cardiovascular,and the growing attention on type 2 diabetes led to larger trial shares in China and India.The GU and Oph TAs are at the lower end of trial initiations,and here we see a more diverse landscape,with

82、countries such as Egypt,Iran,and Poland in the mix.GU is the only TA with positive Y-O-Y growth(4%).Table 5:Ranking of top 10 industry trial sponsors by trial volumesOverallCountry2022(rank)2021(rank)China3,405(1)3,795(1)US2,876(2)3,310(2)Spain644(3)768(3)UK609(4)742(4)France593(5)689(7)Germany580(6

83、)693(6)Australia577(7)647(9)Canada560(8)710(5)Japan530(9)664(8)Italy475(10)618(10)Autoimmune/InflammationCountry2022(rank)2021(rank)China400(1)420(1)US397(2)397(2)Germany132(3)145(3)Poland125(4)143(4)Canada122(5)132(5)Spain122(6)120(8)UK110(7)130(6)Japan100(8)106(10)France92(9)109(9)Australia91(10)1

84、26(7)OncologyCountry2022(rank)2021(rank)China1,492(1)1,747(1)US1,256(2)1,434(2)France270(3)322(4)Spain267(4)341(3)Australia226(5)255(6)UK219(6)251(7)Japan207(7)249(8)Italy203(8)269(5)Germany202(9)237(10)South Korea191(10)246(9)16October 2023 Copyright 2023 Citeline,a Norstella company.(Unauthorized

85、photocopying prohibited).2022 Clinical Trials Roundup*Rank data unavailable Source:Trialtrove,June 2023Infectious DiseaseCountry2022(rank)2021(rank)China416(1)457(2)US304(2)475(1)Spain74(3)99(8)India74(3)213(3)Australia72(5)60(18)UK70(6)104(6)Germany60(7)82(12)South Africa59(8)90(11)Japan47(9)98(9)F

86、rance47(9)63*CNSCountry2022(rank)2021(rank)US511(1)550(1)China388(2)407(2)Canada106(3)116(4)Australia102(4)98(5)UK100(5)118(3)France90(6)78(9)Spain87(7)87(7)Japan85(8)87(7)Italy80(9)70(11)Germany77(10)96(6)Poland107(14)80(9)CardiovascularCountry2022(rank)2021(rank)China309(1)389(1)US146(2)178(2)Cana

87、da54(3)70(3)South Korea49(4)52(7)Russia45(5)47(11)UK44(6)58(4)Japan42(7)49(9)France41(8)48(10)Germany41(9)54(5)Spain40(10)50(8)Iran-54(6)Metabolic/EndocrinologyCountry2022(rank)2021(rank)China403(1)395(1)US277(2)333(2)Germany76(3)85(4)UK74(4)91(3)India73(5)54(12)Canada69(6)81(7)France63(7)68(11)Sout

88、h Korea62(8)79(8)Spain62(9)73(9)Australia58(10)72(10)Japan-83(5)Italy-83(5)GenitourinaryCountry2022(rank)2021(rank)China65(1)39(1)US24(2)19(3)Iran13(3)39(1)India11(4)8(4)Egypt9(5)3(12)Germany7(6)8(9)Japan7(7)9(7)Belgium5(8)4(11)Denmark5(9)4(11)France5(10)3(12)OphthalmologyCountry2022(rank)2021(rank)

89、US74(1)83(1)China40(2)42(2)Australia13(3)19(5)Japan10(4)10(17)Spain9(5)22(3)France8(6)16(10)Italy8(6)14(13)Iran7(8)5*Germany7(8)18(6)Poland7(8)18(7)Overall,the geographic survey of trial activities showed relatively calm waters,with the same top 10 countries and minor shifts in the rankings.When we

90、delve into individual TAs,Autoimmune/Inflammation,Onc,and CNS demonstrated stability of the top countries and their rankings.We observed most upsets in ranking and countries within the ID,GU,and Oph TAs.Russias clinical trials had a clear presence in 2021,but the war with Ukraine took that away in 2

91、022.Without an armistice in sight,the momentum of clinical trials in war-affected countries may take some time to rebuild.17October 2023 Copyright 2023 Citeline,a Norstella company.(Unauthorized photocopying prohibited).2022 Clinical Trials RoundupConcluding ThoughtsIn 2022,we saw fewer clinical tri

92、als than the previous year,which boasted a tremendous rebound after the pandemic disruptions.The smaller trials landscape reflects the macroeconomic pressures in the biopharma industry,as many companies struggled to raise capital for R&D and fund clinical trials.The economic downturn and deflation i

93、n China hampered its post-pandemic recovery and,in turn,raised uncertainties about policies and geopolitical risks for global pharma companies to navigate in China.7 This was compounded by considerable acceleration of divestments and US restrictions of foreign investment on sensitive technologies.Ot

94、her allies of the US are expected to follow suit.How do these factors impact the clinical trials landscape?As one of the largest global economies,the health of Chinas market is inextricably linked to the worlds major markets.One example of how biopharma was affected is in cell and gene therapies(CGT

95、s),an area that was booming with market opportunities within China and now faces uncertainties in regulatory reviews and future development prospects.8The US also encountered its own economic pressures with inflation,which rippled across the global economy.With the top two countries experiencing maj

96、or funding cuts and financial hardships,these factors inevitably translate into fewer clinical trials.The signing of the Inflation Reduction Act(IRA)in late 2022 obviously would not have impacted the clinical trials landscape yet,but we shall see how it affects innovations and efficiencies of clinic

97、al trials in future editions of our Clinical Trials Roundup.As Medicare exerts pricing pressures on drug companies,there are concerns that the companies may shrink their R&D expenditures,potentially hampering advancement to life-saving therapies down the line.On the upside,this legislation is intend

98、ed to target top-selling drugs with market longevity and may bring about a renewed push to fill the pipeline with new discoveries distinguished by higher vigilance on cost efficiency.This projection remains to be seen within the next decade and is an interesting topic for another white paper.But jus

99、t how can companies bring about better efficiencies in clinical trials amid a harsh economic climate?We have heard advocating voices calling for patient-centric approaches through the adoption of decentralized clinical trials(DCTs)elements into protocol design and integration of real-world data/evid

100、ence(RWD/RWE)in strategic R&D.Conversations on DCTs and RWD had swept across the healthcare industry before the pandemic,but the impetus of COVID-19 certainly boosted their importance in bringing clinical trials into the modern age of generative artificial intelligence(AI)and data science technology

101、.The major benefits of DCTs are their patient-centric attributes,such as greater patient flexibility and access,improved enrollment and retention,and enablement of diversity,equity,and inclusion(DE&I).In addition to traditional pharma companies,big-chain pharmacies like CVS,Walgreens,Walmart,and Kro

102、ger have joined the clinical trials business to further enable convenience and diversity in trials.To fully embrace DCT elements in clinical trials,whether it is a fully remote trial or a hybrid model,sponsors are opening 7.Scrip(July 19,2023).More Uncertainty for Biopharma Players as China Economic

103、 Growth Falters.8.Pink Sheet(Sept.26,2022).Cell,Gene Therapy a No-entry Zone?EU Group Seeks Clarification over China Regs.18October 2023 Copyright 2023 Citeline,a Norstella company.(Unauthorized photocopying prohibited).2022 Clinical Trials Roundupdialogues with policymakers on roadblocks such as da

104、ta integration,variability in data collection,trust and harmonization from the regulatory agencies,and partnerships with local communities.These are just a few factors that stand in the way of a strong ROI.The complexity of balancing the benefits and pain points led to a slow adoption(3.4%in 2022)of

105、 clinical trials incorporating DCT elements,as analyzed in Citelines publication,Unlocking the Potential of Decentralized Clinical Trials.9 Steps moving forward on DCT adoption will likely require more open dialogues among sponsors,regulatory bodies,trial sites or local HCPs,and patients.Similarly,t

106、he use of RWD/RWE can expedite accessibility to critical data that enables expanded patient populations for clinical trial recruitment.RWD leverages data from multiple sources including electronic health records(EHR),medical claims data,patient registries,and other digital health technologies that i

107、nform on health status.Even clinical practice with products under emergency use authorization(EUA)is considered within the scope of RWD,as the FDA clarified in its final guidance last month.10 The race is already on for biopharmaceuticals and trial sponsors to seize the opportunities in drug develop

108、ment and clinical trials.Other advances in data science,like generative AI and machine learning(ML),are also creeping their way into the development of drug and biological products.This could potentially impact the benefit-risk assessment of a medical product.The examples of AI/ML span a wide spectr

109、um,from smart pillboxes and electronic medication tracking for protocol adherence to safety surveillance and compliance monitoring.One of the main challenges in AI/ML application is the barrier to transparency,due to the lack of well-defined models for training and validation.Another valid concern i

110、n this area is the precarious balance between transparency and protection of intellectual property,due to the proprietary details within the training sets and the underlying algorithms.However,AI/ML can help sponsors meet diversity goals in clinical trials,especially now that the FDA requires the in

111、clusion of diversity action plans in select late-stage trials.ML tools can potentially evaluate social determinants in claims data to predict diverse enrollment,resulting in higher chances of recruiting historically excluded populations.11 The nature of the AI/ML approach effectively paves the way t

112、o enable DCTs.Despite the challenges ahead,one thing is clear:AI/ML,RWD/RWE,and DCTs are here to stay,and it will take time to see how they shape and modernize clinical trials.Trial protocol design is another way to incorporate efficiency,where we observed an increasing trend of platform trials stud

113、ies that investigate more than one drug intervention and/or indication.This approach was primarily adopted in oncology and COVID-19 trials during the pandemic years(202021).The benefits of having a master protocol in platform trials include the advantage of dosing pre-evaluation and common control a

114、rms,which allow for flexibility of novel combinations and biomarker development.12 Another emerging trend in clinical trials is the use of synthetic control arms or historical controls,as these can potentially replace placebo controls in trials for life-threatening diseases.In a post-pandemic world,

115、the evolving 9.Citeline(2023).Unlocking the Potential of Decentralized Clinical Trials10.Pink Sheet(Sept.10,2023).Real-World Data Can Include EUA Observations,US FDA Final Guidance Says.11.In Vivo(Aug.16,2023).BioBytes:Machine Learning Could Improve Clinical Trial Diversity.12.Scrip(Feb.28,2023).Scr

116、ip AsksWhat Does 2023 Hold for Biopharma?Part 7:Clinical Trial Trends.19October 2023 Copyright 2023 Citeline,a Norstella company.(Unauthorized photocopying prohibited).2022 Clinical Trials Rounduplandscape of clinical trials just got more complex.The year 2022 was one of many adjustments given the i

117、ncidence of lower trial initiations.The healthcare industry endured the impacts of geopolitical conflicts,curbed growth in China,and stagflation in major markets.Regardless of these challenges,we find glimpses of stability in TAs,trial phases,top diseases,major players,and geographic distributions.T

118、he impact of the IRA on the biopharma industry still looms around the corner,and FDA mandates on diversity action plans complicate things.But the resilience shown through the pandemic is not fading;rather,it is preparing the industry to encounter future waves of challenges.Open discussions on ways t

119、o enable modernization in the context of DCTs,RWD/RWE,and innovative trial designs are works in progress to usher in a new age for clinical trials.Heidi ChenAssociate Director,Research&Commercial ServicesHeidi Chen supports client engagement with Citeline content through a deep understanding of cust

120、omer business needs,seasoned expertise in proprietary platforms,market analysis,and commercial activities.She oversees Citelines production of customized Platinum Datapacks across hot topics facing the industry today.Heidi draws from her pharmaceutical industry experience as a drug discovery scienti

121、st and over two decades of therapy-area expertise in CNS,immunology,and ophthalmology.Within this remit,she collaborates across various functional groups within Citeline,and contributes towards projects such as consulting engagements,webinars,and thought leadership white papers.Meet the AuthorCopyri

122、ght 2023 Citeline,a Norstella company.Pharma Intelligence UK Limited is a company registered in England and Wales with company number 13787459 whose registered office is 3 More London Riverside,London SE1 2AQ.Citeline,a Norstella company,powers a full suite of complementary business intelligence off

123、erings to meet the evolving needs of life science professionals to accelerate the connection of treatments to patients and patients to treatments.These patient-focused solutions and services deliver and analyze data used to drive clinical,commercial,and regulatory-related decisions and create real-w

124、orld opportunities for growth.Our global teams of analysts,journalists,and consultants keep their fingers on the pulse of the pharmaceutical,biomedical,and medtech industries,covering it all with expert insights:key diseases,clinical trials,drug R&D and approvals,market forecasts,and more.For more information on one of the worlds most trusted life science partners,visit C